Kathy Giusti story

It has been nine years since Kathy Giusti was diagnosed with multiple myeloma, an incurable form of blood cancer with one of the lowest five-year survival rates of any cancer.

"My daughter was 1 at the time. My goal was to see her go to kindergarten," Giusti recalled.

This month, she saw her daughter off to sixth grade.

"It's a nice story," said Giusti, a New Canaan resident who since has also given birth to a boy.

Scientists and cancer survivors say more people are alive today because of the work Giusti has done since 1996. The former pharmaceutical executive and her twin sister, Karen Andrews, have started not one but two separate nonprofit organizations dedicated to jump-starting research into multiple myeloma. The Multiple Myeloma Research Foundation has raised $47 million for research, and the Multiple Myeloma Research Consortium has forged a scientific alliance of seven major cancer research institutions and created the first multiple myeloma tissue bank.

"Kathy and her sister Karen have turned adversity into an opportunity for multiple myeloma patients worldwide and gave them hope for a better life," said Dr. Ken Anderson, chief of the division of hematologic neoplasia at the Dana Farber Cancer Institute in Boston. Anderson serves on the boards of both the foundation and consortium.

When Giusti was first diagnosed in 1996, the only treatment options available for the approximately 14,000 people diagnosed with multiple myeloma each year in the United States were chemotherapy and stem cell transplantation, which is designed to replace blood cells destroyed during chemo.

Those treatments often bought patients a reprieve from cancer, but there remains no cure.

"It is hard enough to be diagnosed with a cancer, but it was unbelievable to be diagnosed by one that is uniformly fatal," Guisti said.

She took a survey of research activity at drug companies and academic institutions and found that little work was being done on the disease.

"I told my sister that this looks pretty dismal," she said.

Giusti and Andrews set about to change that outlook. In 1998, they formed the Multiple Myeloma Research Foundation to raise money and promote awareness of the disease. They went a step further and took an active role in promoting the development of promising research at both drug companies and federal regulatory agencies.

The creation of the foundation coincided with the discovery that a notorious drug called thalidomide seemed to halt the progression of blood cancer in many patients. Thalidomide was taken by many pregnant women in the 1960s to ward off morning sickness. After the drug was discovered to cause serious birth defects, it was approved only to treat patients with leprosy. Giusti said the foundation began educating both the public and physicians about the potential benefits of the drug, which is now commonly prescribed for multiple myeloma patients who have had a recurrence.

Thalidomide, however, is not a cure. So the foundation has aggressively backed the development of two new multiple myeloma drugs. Velcade, designed for patients whose cancer has become resistant to treatment, was approved in 2003. Revimid, an immune-system booster, is now in Phase III trials.

"We went from no drugs to three since we started," Giusti said.

One multiple myeloma patient who has benefited from the new treatment options is the Connecticut weather forecaster Mel Goldstein. Like Giusti, he was diagnosed in 1996. He was one of the first patients in the country to be given thalidomide for his cancer.

"I can't speak highly enough [about] progress that has been made in the field. It occurred as the foundation was raising funds and awareness of the disease and getting drug companies to develop new treatments," Goldstein said.

Linking drug companies and academic centers developing promising new treatments was one of Giusti's goals in 2004, when she formed the Multiple Myeloma Research Consortium. One of the consortium's initiatives was to create the first multiple myeloma tissue bank. New drugs under investigation can be tested on tissue from multiple myeloma patients. The hope is that successful tests will prompt pharmaceutical companies to develop the new drug, Giusti said.

She also has announced plans to have scientists map the genome of multiple myeloma, with an aim to identify key genetic markers in the development of the disease and additional drug development targets.

"I never lose my sense of urgency," said Giusti, who, like Goldstein and an estimated 45,000 myeloma patients, still live under a death sentence from the disease. "We run this like a Fortune 100 company. We don't mess around here."

By William Hathaway, Courant Staff Writer

Copyright 2005, Hartford Courant